While pediatric trial participation is vital in discovering and understanding new treatments, the number of patients (as well as the number of available trials) remains relatively low. According to the World Health Organization (WHO), only 16.7% of trials on the organization’s portal involve pediatric patients (the same number is 12% on ClinicalTrials.gov), though children contribute to nearly 60% of the total disease burden of studied conditions.
In an effort to better understand and combat the problem. Parexel and the Center for Information and Study on Clinical Research Participation (CISCRP) joined forces to conduct a survey of pediatric patients and parents. Outsourcing-Pharma (OSP) spoke with two professionals about the survey:
- Shipra Patel (SP) senior medical director and global head of pediatrics with Parexel
- Annick de Bruin (ADB), senior director of research services with CISCRP
The two experts shared an overview of the survey and what it revealed about pediatric clinical research.
OSP: Could you please share a description of CISCRP—who you are, what you do, key services/projects and how your mission sets you apart from other organizations?
ADB: CISCRP is a non-profit organization founded in 2003. CISCRP is dedicated to educating and engaging critical stakeholders -- the public, patients, the healthcare community, policy makers and the media -- as partners in the clinical research process.
CISCRP provides a variety of educational resources, programs and services including print and digital materials; media outreach and awareness campaigns, live educational events; study volunteer appreciation programs; patient advisory boards; custom research assessing patient health journeys and study volunteer experiences; and plain language clinical trial results summaries. CISCRP also provides patients with a free service to locate active clinical trials.
OSP: Can you please summarize the survey, and why you chose to conduct it?
ADB: Every two years, CISCRP conducts the Perceptions & Insights Survey – an online, global assessment of public and patient perceptions, motivations, and experiences with clinical research participation. The goal is to monitor trends and identify opportunities to better inform and engage the public and patients as stakeholders and partners in the clinical research enterprise.
In an effort to build off this work, Parexel engaged CISCRP to design a survey specifically for parents and their children in order to better understand perceptions and experiences related to pediatric clinical trials. Specifically, the goal of this survey was to gain insights into general perceptions of pediatric trials, preferred channels of communication, key information parents and their children would want about pediatric clinical trials when deciding whether to participate, as well as current and past experiences related to pediatric clinical trial participation.
During April 2020, CISCRP conducted an online, US-based survey of 500 parents and their children. The survey instrument was based in part on questions posed in past Perceptions & Insights studies, with modified language for child respondents. Parents were asked to complete the adult section of the survey, then ask their children to complete the child section.
The insights we’ve gleaned will help the industry at large design more patient-friendly trials that better address the needs of patients and families, while also improving how we communicate with parents and children about the reasons for participating and how they align with parent/child motivations.
OSP: What do you believe is the most notable finding in the survey? Were there any surprises?
ADB: Not unexpectedly, parents reported highly burdensome experiences and high levels of disruption to their daily routine when participating in a clinical trial. Children also reported burdensome experiences, although most children who had participated in a clinical trial indicated that their experience was better than they anticipated it would be.
These findings further reinforce what we’ve learned throughout the pandemic, that decentralized clinical trial approaches such as telehealth visits, home health nurse visits, wearable devices and direct-to-patient drug shipments can really ease the burden of trial participation while also making trials much more accessible to people who might otherwise not be able to join.
Perhaps less expected, child respondents said that the opportunity to hear from other children who have taken part in a study was very important. This finding should be explored further so that we can potentially develop programs in which kids share with other kids what it’s like to be in a trial.
This peer-to-peer interaction is so important for kids and teens, and it’s a really interesting model for raising awareness about clinical trials among kids.
OSP: You noted pediatric trials make up a relatively small portion of global studies. Why is this worth noting, and is it a problem that needs to be addressed?
SP: Each year, hundreds of millions of prescriptions are dispensed to children for the treatment of a wide range of conditions, but the overwhelmingly majority of these medications have not been evaluated in children. This matters because children often have very different metabolic and physiologic responses to medications than adults, so you cannot extrapolate from adult studies to infer how a medication will affect children.
It's critical that we engage more parents and children to participate in clinical trials so that we fully understand the benefits, risks, and side effects of drugs in children. Without evidence from pediatric trials, pediatricians must rely on adult studies or anecdotal data to make treatment decisions about using drugs off-label in individual patients.
In addition, there are many unmet medical needs for children and adolescents because therapies are not tailored to address their unique conditions and needs.
OSP: What are some of the ways industry professionals can help ensure researchers are taking on an appropriate number of pediatric trials?
SP: It is important for industry professionals to collaborate with disease advocacy groups and foundations to provide these groups with detailed information about their clinical trials. In turn, disease associations can share information about actively recruiting trials with both patients and community physicians, who may either refer patients to the trial or decide to become an investigative site.
The CISCRP survey showed that parents trust their child’s pediatrician’s recommendation the most when deciding whether to enroll their child in a clinical trial, so it’s imperative that pediatricians are well informed about local trials for a variety of conditions. Industry professionals can also discuss their trials with key opinion leaders, who can disseminate information about these trials to their physician communities.
Lastly, it’s important to capitalize on the heightened awareness of clinical research that the pandemic has fostered. The public now has a greater appreciation of the necessity of clinical research and the vital role in plays in developing and testing new medications. This new awareness should serve as the catalyst for developing campaigns that further expand this knowledge and encourage more people to participate in clinical trials.
OSP: What role do parent advocates play in effecting improvements in the pediatric trial field?
SP: When a child is enrolled in a trial, in essence the entire family is enrolled, because parents and siblings – and sometimes extended family members – have to coordinate their lives around the trial requirements. This can place a significant burden on families who are juggling jobs, home responsibilities, school and other children.
Parents who have been through the experience of a pediatric trial and are now serving as patient advocates bring a real-world perspective to the table when sharing the true impact of trial participation on families. This feedback is invaluable both for families who are considering trial participation and for drug developers who are designing pediatric trials. They need to understand potential obstacles and how to ease them, from the smallest details such as ensuring Wi-fi at a study site to the big issues such as transportation and scheduling.
For families of children with rare diseases, participating in a clinical trial can often mean moving the family from one city or state to another, or possibly to another country, which is overwhelming and disruptive to the entire family. Parent advocates can help educate other parents about what to expect, what to prepare for, and how to best navigate the dynamics associated with such a momentous change.
Parent advocates can also provide critical support for parents and kids who are feeling isolated or alone with their disease. Hearing from another family that’s been through the experience of the disease and a clinical trial is both comforting and reassuring at a time when everything feels uncharted and unknown.